FDA Approves Muscular Dystrophy Drug That Patients Lobbied For

The New York Times, September 19, 2016. The Food and Drug Administration approved the first drug to treat patients with the most common childhood form of muscular dystrophy. The agency’s approval went against the recommendation of FDA experts and the agency’s independent Advisory Committee. “The agency has set a dangerous precedent,” said Diana Zuckerman, president of the National Center for Health Research.

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FDA Grants Accelerated Approval for Sarepta Muscular Dystrophy Drug Eteplirsen

Tech Times, September 20, 2016. The U.S. Food and Drug Administration (FDA) has approved the first drug for treating patients with Duchenne muscular dystrophy. “The agency has set a dangerous precedent,” said National Center for Health Research president Diana Zuckerman. “To prove something works, you have to compare it to something else — a placebo or a treatment. They didn’t do that.”

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FDA Approves First Drug to Treat Rare Form of Muscular Dystrophy

Kaiser Health News, September 19, 2016. The Food and Drug Administration Monday approved the first drug to treat Duchenne muscular dystrophy, a rare and lethal muscle weakening disorder that affects about 15,000 Americans. Diana Zuckerman, president of the National Center for Health Research, said “it sets a dangerous precedent if the FDA is going to start approving drugs that aren’t compared to anything.”

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